Gene-edited stem cells: a lasting fix for sickle cell?

NCT ID NCT06155500

Ongoing Disease control Sponsor: Novartis Pharmaceuticals Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jul 29, 2026 · Updated 3 times

Summary

This study follows 4 people with sickle cell disease who previously received a one-time treatment of their own gene-edited stem cells (OTQ923). Researchers are checking for delayed side effects, such as new cancers or blood disorders, and measuring how long fetal hemoglobin production lasts. The goal is to see if the treatment remains safe and effective over time.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: OTQ923 (gene-edited stem cells)
What this could lead to: If successful, this could show that a single treatment with gene-edited stem cells provides lasting control of sickle cell disease symptoms.
What could go wrong: This is a very small, early-phase follow-up study with only 4 participants, so results may not apply broadly. Long-term risks like new cancers or blood disorders are still being checked.

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Conditions

The condition(s) this trial relates to.

sickle cell disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

SICKLE CELL DISEASE

Contacts and locations

Locations

Memorial Sloan Kettering Cancer Ctr

New York, New York, 10065, United States
St Jude Childrens Research Hospital

Memphis, Tennessee, 38105, United States
University of Chicago

Chicago, Illinois, 60637, United States

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